Real-World Data Reveal High Rates of Trientine Tetrahydrochloride Adherence for Wilson Disease

New research is shedding light on high rates of treatment adherence and low discontinuation rates among patients with Wilson disease treated with trientine tetrahydrochloride (TETA-4HCl, Cuvrior).1

Data from the real-world analysis were presented at the American Association for the Study of Liver Diseases (AASLD) The Liver Meeting 2025 by Veronica Tomor, PharmD, BCPS, a clinical pharmacist and Associate Director Medical and Scientific Affairs at Orphalan, and highlight high treatment adherence with minimal interruption in therapy as described with a gap of 2.7 days per shipment and a low overall discontinuation rate of 17%.1

In May 2022, the US Food and Drug Administration approved Orphalan’s trientine tetrahydrochloride for the treatment of adult patients with stable Wilson disease who are de-coppered and tolerant to penicillamine. The treatment later became commercially available in 2023, offering a novel, effective, long-term treatment option for a patient population that has historically faced limited therapeutic choices.2

“[Wilson disease] prognosis improves significantly with early diagnosis, effective copper control, and sustained lifelong therapy,” Tomor and colleagues wrote.1 “Adherence to treatment is essential for achieving optimal clinical outcomes; however, it remains a significant challenge for patients, and poor adherence is associated with disease progression.”

To assess treatment adherence to TETA-4HCI, investigators conducted a retrospective analysis of data from a pharmacy database for patients who were prescribed and ≥ 1 shipment of TETA-4HCl between May 2023 and December 2024. They collected and defined the following variables:

• Proportion of Days Covered (PDC), defined as the proportion of days where patients actively have medication on hand over a given time with a minimum time for calculation of 180 days
• Gap days, defined as the number of days a patient does not have medication on hand based on shipment history
• Treatment discontinuation with reasons
• Demographics
• Prescriber data

During the study period, 142 patients were identified and included in the demographics. The majority (55%) were male with an overall mean age of 39 (standard deviation [SD], 15.7) years and were prescribed TETA-4HCl. Among the cohort, 45 patients (32%) traveled out of state to consult with their prescriber.1

Patient payer segmentation was 72% commercial, 15% Medicare, 10% Medicaid, and 3% uninsured. Prescribers were predominantly hepatologists and gastroenterologists at 64% and 23% respectively.1

A total of 46% of patients indicated a history of treatment with > 1 therapy, the most frequently reported being trientine hydrochloride capsules, zinc salts, and penicillamine.1

A total of 24 patients discontinued therapy, 4 of which were due to an adverse event. Among the cohort, 93 patients met the 180-day threshold for PDC calculation and 126 were included in the gap day calculation. The mean PDC was 84 (SD, 24) %, with an average gap of 2.7 days per shipment.1

“In this real-world analysis of patients with Wilson disease receiving TETA-4HCl through a single-source specialty pharmacy, adherence was consistently high with minimal interruption in therapy as described with a gap of 2.7 days per shipment and a low overall discontinuation rate of 17%,” investigators concluded.1 “The extent to which single-source pharmacy distribution mitigates barriers to access, reduces treatment burden, and facilitates adherence warrants further investigation.”

References

1. Tomor V, Heise J, Dockery C, et al. Medication Adherence in Wilson Disease Patients Treated with Trientine Tetrahydrochloride Supplied by a Single Source Specialty Pharmacy. Presented at the American Association for the Study of Liver Diseases (AASLD) The Liver Meeting 2025. Washington, DC. November 7-11, 2025.

2. Orphalan. Orphalan announces FDA approval of Cuvrior™ for the treatment of adult patients with stable Wilson’s disease who are de-coppered and tolerant to penicillamine. May 2, 2022. Accessed November 10, 2025. https://www.orphalan.com/orphalan-announces-fda-approval-of-cuvrior/